This blog is part of our series “Early Funding Paves the Way for Biomedical Innovation,” featuring conversations with leaders at three companies that are working on diagnostic or therapeutic solutions for people with Inflammatory Bowel Disease (IBD). These stories highlight how early-stage investments accelerated their novel ideas and how our ongoing partnership has supported their evolving work.
Below are highlights from our conversation with Julie Saiki, PhD, Co-Founder and Chief Operating Officer for Azora Therapeutics, which is a clinical-stage biopharmaceutical company developing an alternative to immuno-suppressive therapies.
How do your innovations address gaps in managing and treating IBD?
Julie Saiki, PhD: There is a huge unmet need for new drugs for IBD patients who don’t respond to current treatments. Azora Therapeutics’ work began with a botanical extract called indigo naturalis, which showed remarkably promising results for patients with refractory UC [ulcerative colitis]. We discovered the mechanism was through a transcription factor called aryl-hydrocarbon receptor or AhR. It turns on numerous genes involved in immune modulation and barrier function, both of which are critical for ulcerative colitis. An early Stanford study showed us that rectal bleeding appears to resolve pretty quickly, and many of those patients achieved mucosal healing. Once we identified the active moiety, we developed our own novel small molecule (AT177) compound based on the chemistry of this known naturally-occurring molecule.
There is a huge unmet need in the IBD space for new drugs for patients who don’t respond to current treatments.
How is the current landscape impacting your product development?
Julie: In the broader landscape, market trends are moving toward later stage programs. It’s vital to be able to show that our program, despite being early stage, is de-risked from both an efficacy and safety perspective. Changes in public funding and government regulation have also impacted the biotech ecosystem. But we’ve also seen recovery in the private sector, which gives us hope.
Why have you focused your work on IBD?
Julie: While a graduate student in musicology at Stanford, I was diagnosed with ulcerative colitis [UC]. I wasn’t responding to standard of care, so I tried alternatives, including diet, and stumbled upon indigo naturalis. I had a quite dramatic response. I took this botanical and the next day my rectal bleeding stopped and all my symptoms went away. It immediately turned my life around. My growing interest in drug discovery led me to SPARK at Stanford. After sharing my experience, SPARK supported this as a research project. I ran a small early Phase 1b clinical trial with the extract in 11 patients with refractory UC. We saw great results. It was this robust response in a hard-to-treat patient population that inspired me to start Azora.
What was the role of early funding in your product development?
Julie: Early funding from the Rainin Foundation built on our prior research and helped us reach three pre-critical milestones that de-risked the program from a safety perspective. One was early tox studies on our compound. The second milestone was pharmacokinetic and pharmacodynamic studies with our drug product. The third was refining the formulation for use in clinical study. Once you’ve hit these early-stage milestones, other investors become more comfortable with the program.
Early funding from the Rainin Foundation helped us reach three pre-critical milestones.
What comes next in your development of this technology?
Julie: We see a lot of positives in the data that reinforce our hypothesis that this could be very effective in patients. We’re completing an investigational new drug application package required for FDA approval of a Phase I study. The milestones we achieved with Rainin Foundation funding are pivotal to getting to that Phase I study milestone, and to our discussions with investors and potential partners. Our goal is for Azora to rapidly advance this new discovery so that it can help patients.
What message do you have for the field?
Julie: As an IBD patient, I recognize how hard it is to live with this disease. But I’m thankful to the researchers and clinicians who continue to pursue new, novel treatments to help patients. And I’m inspired by patients who responded to my publication. They’re eager to learn more and excited about the work we’re doing. Funders like the Rainin Foundation are vital for this field, especially in light of reductions in government financing for research. The [Rainin Foundation] community is awesome. I’ve met amazing researchers through their Symposium, where I’ve found a fruitful exchange of ideas and research.
I’m thankful to the researchers and clinicians who continue to pursue new, novel treatments to help patients.
